Are Uncontrolled Clinical Studies Ever Justified?

To the Editor: The publication in the June 5 and 19 issues of two therapeutic studies, by Antman and Dzau and their colleagues, that lack control groups raises serious questions about the conduct of such trials. Both articles are written as though they provided strong evidence for the effectiveness of a new drug for a life-threatening condition, but in fact both are collections of anecdotal experience, uncontrolled and unblinded. Both papers acknowledge this important defect with a line or two in the Discussion, but obviously neither the investigators nor the reviewers thought that the conclusions were compromised. The use of. . .